布仑索替布在支气管扩张症患者中的里程碑式3期ASPEN研究的新亚组分析与阳性总体试验人群结果一致-动脉网

New Subgroup Analyses from Landmark Phase 3 ASPEN Study of Brensocatib in Patients with Bronchiectasis Demonstrate Consistency with Positive Overall Trial Population Results

CISION 等信源发布 2024-10-09 04:05

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—Data Presented at the CHEST 2024 Annual Meeting—

-2024年胸部年会上提供的数据-

BRIDGEWATER, N.J., Oct. 8, 2024 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today presented positive late-breaking subgroup data from the Phase 3 ASPEN study, a global, randomized, double-blind, placebo-controlled Phase 3 study to assess the efficacy, safety, and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis.

新泽西州布里奇沃特（BRIDGEWATER），2024年10月8日/PRNewswire/--Insmed Incorporated（Nasdaq:INSM），一家以人为本的全球生物制药公司，致力于提供一流和最佳的治疗方法来改变面临严重疾病的患者的生活，今天提供了来自ASPEN 3期研究的积极的晚期亚组数据，该研究是一项全球性，随机，双盲，安慰剂对照的3期研究，旨在评估brensocatib在非囊性纤维化支气管扩张患者中的疗效，安全性和耐受性。

Posters with the details from these subgroup analyses can be found HERE. These data were presented at the CHEST 2024 Annual Meeting, held October 6-9 in Boston, along with several other presentations from across Insmed's respiratory portfolio..

有这些亚组分析细节的海报可以在这里找到。这些数据在10月6日至9日于波士顿举行的2024年胸部年会上发表，以及Insmed呼吸组合的其他几篇演讲。。

'The positive results from the landmark Phase 3 ASPEN study have generated tremendous excitement among physicians and patients alike, providing hope for a much-needed approved treatment for bronchiectasis. Given the heterogeneity of this disease, it is striking that these new sub-analyses show a consistent positive impact on the rate of exacerbations across the majority of prespecified subgroups,' said lead study investigator James Chalmers, MBChB, Ph.D., Professor and Consultant Respiratory Physician at the School of Medicine, University of Dundee, UK.

“具有里程碑意义的ASPEN 3期研究的积极结果在医生和患者中产生了巨大的兴奋，为急需的支气管扩张批准治疗提供了希望。“鉴于这种疾病的异质性，令人惊讶的是，这些新的亚组分析显示，在大多数预先指定的亚组中，对恶化率有持续的积极影响，”英国邓迪大学医学院首席研究者、MBChB博士、教授兼呼吸科顾问医师詹姆斯·查尔默斯（JamesChalmers）说。

'It is particularly exciting to see that, consistent with the overall trial analysis, brensocatib 25 mg was able to slow lung function decline as measured by FEV1 for all prespecified subgroups, further underscoring the potential impact this investigational treatment candidate may have on a diverse range of patients if approved.'.

“特别令人兴奋的是，与总体试验分析一致，brensocatib 25 mg能够减缓所有预先指定亚组的FEV1测量的肺功能下降，进一步强调了这一研究治疗候选者可能对多种患者产生的潜在影响，如果批准的话。”。

Consistent with positive results for the overall ASPEN population, the annualized rate of pulmonary exacerbations favored brensocatib at both the 10 mg and 25 mg doses over placebo for almost all subgroups. In a separate analysis, least squares mean difference for brensocatib 25 mg demonstrated a reduced decline in post-bronchodilator FEV1 at Week 52 versus placebo for all prespecified subgroups.

与整个白杨种群的阳性结果一致，几乎所有亚组的肺部急性加重年化率均优于安慰剂10 mg和25 mg剂量的brensocatib。在另一项分析中，brensocatib 25 mg的最小二乘平均差显示，与安慰剂相比，所有预先指定的亚组在第52周时支气管扩张剂后FEV1的下降幅度均有所降低。

Results for patients treated with brensocatib 10 mg among subgroups were aligned with the overall trial population results..

亚组中使用brensocatib 10 mg治疗的患者的结果与总体试验人群结果一致。。

Brensocatib was well-tolerated in the study. Treatment-emergent adverse events (TEAEs) occurring in at least 5.0% of patients treated with either dose of brensocatib and more frequently than in placebo were COVID-19 (15.8%, 20.9%, 15.8%), nasopharyngitis (7.7%, 6.3%, 7.6%), cough (7.0%, 6.1%, 6.4%), and headache (6.7%, 8.5%, and 6.9%) for brensocatib 10 mg, brensocatib 25 mg, and placebo, respectively..

Brensocatib在研究中耐受性良好。在接受任一剂量brensocatib治疗且比安慰剂更频繁的患者中，至少有5.0%的患者出现治疗紧急不良事件（TEAE），其中COVID-19（15.8%，20.9%，15.8%），鼻咽炎（7.7%，6.3%，7.6%），咳嗽（7.0%，6.1%，6.4%）和头痛（6.7%，8.5%和6.9%）分别为brensocatib 10 mg，brensocatib 25 mg和安慰剂。。

'We are incredibly excited to present subgroup findings from the ASPEN study at CHEST 2024, building on the positive results we have already shared with the bronchiectasis community,' said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. 'The consistency of efficacy observed across most subgroups, particularly at the higher dose, further supports our belief that brensocatib has the potential to transform the treatment landscape for patients with bronchiectasis.

Insmed首席医学官、MBA医学博士马蒂娜·弗拉默（MartinaFlammer）说：“我们非常高兴能在2024年胸围（CHEST 2024）上展示ASPEN研究的亚组结果，这是基于我们已经与支气管扩张社区分享的积极结果。”在大多数亚组中观察到的疗效的一致性，特别是在较高剂量下，进一步支持了我们的观点，即brensocatib有可能改变支气管扩张患者的治疗前景。

We eagerly look forward to filing a new drug application for brensocatib in the U.S., followed by Europe and Japan, in hopes of delivering the first approved therapy for patients affected by this serious and difficult-to-treat disease.'.

我们热切期待着在美国、欧洲和日本提交brensocatib新药申请，希望为受这种严重且难以治疗的疾病影响的患者提供首个批准的治疗。”。

Insmed plans to file a New Drug Application with the U.S. Food and Drug Administration for brensocatib in patients with bronchiectasis in the fourth quarter of 2024. Pending regulatory approvals, Insmed anticipates a U.S. launch for brensocatib in mid-2025 followed by launches in Europe and Japan in the first half of 2026.

Insmed计划于2024年第四季度向美国食品和药物管理局提交一份针对支气管扩张患者的brensocatib新药申请。在获得监管部门批准之前，Insmed预计brensocatib将于2025年年中在美国上市，然后于2026年上半年在欧洲和日本上市。

If approved, brensocatib would be the first approved treatment for patients with bronchiectasis as well as the first approved dipeptidyl peptidase 1 (DPP1) inhibitor—a new mechanism of action with the potential to address a range of neutrophil-mediated diseases..

如果获得批准，brensocatib将是第一个被批准用于支气管扩张患者的治疗方法，也是第一个被批准的二肽基肽酶1（DPP1）抑制剂，这是一种新的作用机制，有可能解决一系列中性粒细胞介导的疾病。。

About ASPENAs part of the ASPEN study's conduct, more than 460 trial sites were engaged in nearly 40 countries. After excluding sites that did not enroll any patients and all sites in Ukraine, the total number of active sites in ASPEN was 391 sites in 35 countries. Adult patients (ages 18 to 85 years) were randomized 1:1:1 and adolescent patients (ages 12 to <18 years) were randomized 2:2:1 for treatment with brensocatib 10 mg, brensocatib 25 mg, or placebo once daily for 52 weeks, followed by 4 weeks off treatment.

关于ASPENAs作为ASPEN研究的一部分，在近40个国家有460多个试验点参与。在排除未招募任何患者的网站和乌克兰的所有网站后，ASPEN的活动网站总数为35个国家的391个网站。成年患者（年龄18至85岁）以1:1:1的比例随机分组，青少年患者（年龄12至18岁）以2:2:1的比例随机分组，每天一次用brensocatib 10 mg，brensocatib 25 mg或安慰剂治疗52周，然后停止治疗4周。

The primary efficacy analysis included data from 1,680 adult patients and 41 adolescent patients..

主要疗效分析包括1680名成年患者和41名青少年患者的数据。。

About BrensocatibBrensocatib is a small molecule, oral, reversible inhibitor DPP1 being developed by Insmed for the treatment of patients with bronchiectasis, CRSsNP, and other neutrophil-mediated diseases. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow.

关于BrensocatibBrensocatib是由Insmed开发的一种小分子口服可逆抑制剂DPP1，用于治疗支气管扩张症，CRSsNP和其他中性粒细胞介导的疾病。DPP1是一种酶，负责激活中性粒细胞丝氨酸蛋白酶（NSPs），如中性粒细胞弹性蛋白酶，当它们在骨髓中形成时。

Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs.

中性粒细胞是最常见的白细胞类型，在病原体破坏和炎症介导中起着至关重要的作用。在慢性炎症性肺病中，嗜中性粒细胞在气道中积聚并导致过度活跃的NSP，导致肺部破坏和炎症。Brensocatib可以通过抑制DPP1及其NSP的激活来降低炎症性疾病如支气管扩张的破坏作用。

Brensocatib is an investigational drug product that has not been approved for any indication in any jurisdiction..

Brensocatib是一种研究药物，尚未在任何管辖区批准用于任何适应症。。

About InsmedInsmed Incorporated is a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases. The Company is advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving patient communities where the need is greatest.

关于InsmedInsmed Incorporated是一家以人为本的全球生物制药公司，致力于提供一流和最佳的治疗方法，以改变面临严重疾病的患者的生活。该公司正在推进批准和中晚期研究药物的多样化组合，以及专注于服务需求最大的患者社区的尖端药物发现。

Insmed's most advanced programs are in pulmonary and inﬂammatory conditions, including a therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company's early-stage research programs encompass a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue..

Insmed最先进的项目是肺部和炎症性疾病，包括美国，欧洲和日本批准的治疗慢性衰弱性肺病的疗法。该公司的早期研究计划涵盖了广泛的技术和模式，包括基因治疗、人工智能驱动的蛋白质工程、蛋白质制造、RNA末端连接和合成拯救。。

Headquartered in Bridgewater, New Jersey, Insmed has offices and research locations throughout the United States, Europe, and Japan. Insmed is proud to be recognized as one of the best employers in the biopharmaceutical industry, including spending three consecutive years as the No. 1 Science Top Employer.

Insmed总部位于新泽西州布里奇沃特，在美国、欧洲和日本设有办事处和研究地点。Insmed很自豪被公认为生物制药行业最好的雇主之一，包括连续三年成为第一大科学顶级雇主。

Visit www.Insmed.com to learn more..

Forward-looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. 'Forward-looking statements,' as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties.

前瞻性声明本新闻稿包含涉及重大风险和不确定性的前瞻性声明。”前瞻性陈述，正如1995年《私人证券诉讼改革法案》中所定义的那样，是指非历史事实的陈述，涉及许多风险和不确定性。

Words herein such as 'may,' 'will,' 'should,' 'could,' 'would,' 'expects,' 'plans,' 'anticipates,' 'believes,' 'estimates,' 'projects,' 'predicts,' 'intends,' 'potential,' 'continues,' and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements..

本文中的词语，如“可能”、“将会”、“应该”、“可能”、“将会”、“期望”、“计划”、“预期”、“相信”、“估计”、“项目”、“预测”、“打算”、“潜在”、“继续”，以及类似的表达（以及引用未来事件、条件或情况的其他词语或表达）可能会识别前瞻性陈述。。

The forward-looking statements in this press release are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timings discussed, projected, anticipated or indicated in any forward-looking statements.

本新闻稿中的前瞻性声明基于公司当前的期望和信念，涉及已知和未知的风险、不确定性和其他因素，这些因素可能导致公司的实际业绩、业绩和成就以及某些事件的发生时间与任何前瞻性声明中讨论、预测、预期或指示的业绩、业绩、成就或时间有重大差异。

Such risks, uncertainties and other factors include, among others, the following: the risk that the full data set from the ASPEN study or data generated in further clinical trials of brensocatib will not be consistent with the topline results of the ASPEN study or any additional results of the ASPEN study; failure to obtain, or delays in obtaining, regulatory approvals for brensocatib in the U.S., Europe or Japan; failure to successfully commercialize brensocatib, if approved by applicable regulatory authorities, in the U.S., Europe or Japan, or to maintain U.S., European or Japanese approval for brensocatib once approved; uncertainties in the degree of market acceptance of brensocatib by physicians, patients, third-party payors and others in the healthcare community; inaccuracies in the Company's estimates of the size of the potential markets for brensocatib or in data the Company has used to identify physicians; expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; inability of the Company, Esteve Pharmaceuticals, S.A., Thermo Fisher Scientific, Inc.

这些风险、不确定性和其他因素包括以下因素：ASPEN研究的完整数据集或brensocatib进一步临床试验产生的数据与ASPEN研究的主要结果或ASPEN研究的任何其他结果不一致的风险；未能获得或延迟获得美国、欧洲或日本brensocatib的监管批准；如果获得美国、欧洲或日本相关监管机构的批准，未能成功将brensocatib商业化，或者一旦获得批准，未能保持美国、欧洲或日本对brensocatib的批准；医生，患者，第三方付款人和医疗保健界其他人对brensocatib的市场接受程度的不确定性；公司对brensocatib潜在市场规模的估计不准确，或公司用于识别医生的数据不准确；预期患者摄取率，预期治疗持续时间或预期患者依从性或停药率；公司无能力，Esteve Pharmaceuticals，S.A.，Thermo Fisher Scientific，Inc。

or the Company's other third-party manufacturers to comply with regulatory requirements related to brensocatib; the Company's inability to obtain adequate reimbursement from government or third-party payors for .

；公司无法从政府或第三方付款人处获得足够的报销。

The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company's forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future.

公司可能无法实际实现公司前瞻性声明所示的结果、计划、意图或期望，因为就其性质而言，前瞻性声明涉及风险和不确定性，因为它们与事件有关，取决于未来可能发生或不可能发生的情况。

For additional information about the risks and uncertainties that may affect the Company's business, please see the factors discussed in Item 1A, 'Risk Factors,' in the Company's Annual Report on Form 10-K for the year ended December 31, 2023 and any subsequent Company filings with the Securities and Exchange Commission (SEC)..

有关可能影响公司业务的风险和不确定性的更多信息，请参阅公司截至2023年12月31日的年度报告表10-K中第1A项“风险因素”中讨论的因素，以及公司随后向美国证券交易委员会（SEC）提交的任何文件。。

The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements..

该公司提醒读者不要过度依赖任何此类前瞻性声明，这些声明仅在本新闻稿发布之日起生效。除法律和美国证券交易委员会规则明确要求外，公司无义务公开更新或修订任何此类声明，以反映任何此类声明所依据的预期或事件、条件或情况的任何变化，或可能影响实际结果与前瞻性声明所述结果不同的可能性。。

Contact:

联系人：

Investors:

投资者：

Bryan DunnExecutive Director, Investor RelationsInsmed(646) 812-4030bryan.dunn@insmed.com

Bryan Dunnexecutive董事，投资者关系医学（646）812-4030bryan.dunn@insmed.com

Media:

媒体：

Mandy FaheyVice President, Corporate CommunicationsInsmed(732) 718-3621amanda.fahey@insmed.com

Mandy FaheyCorporate CommunicationsInsmed副总裁（732）718-3621amanda.fahey@insmed.com

SOURCE Insmed Incorporated